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2 BioStrategics Consulting Ltd THE CLINICAL TRIAL PROTOCOL The clinical trial protocol to test one s product, to confirm or reject a specific hypothesis, is key in the clinical development plan and in the overall development of the product with the goal of moving the science forward and preparing a product for market acceptance. The clinical trial protocol is the experimental blueprint for a clinical study, and every activity and procedure it specifies should contribute to the efficient testing of the central hypothesis being examined. A DETAILED GUIDE TO THE CLINICAL TRIAL PROTOCOL KEY PROTOCOL PROTOCOL GUIDANCE ELEMENTS 1. Key Roles Define institutions Sponsor Principal investigator Investigators Key responsible individuals 2. Product rationale; potential risks and benefits Name of drug Summary of preclinical data Summary of clinical data Relevant background literature Potential risks and benefits Importance of the study 3. Study objectives Definition of the goals of the study. Should define the gathering of data that are absolutely required building blocks of the total registration dossier of the new drug. Several major categories of clinical trial objectives exist: Dose-dependent objectives: o Safety and tolerability o Biological effect, pharmacodynamics, surrogate end points o Absolute efficacy (i.e., versus no treatment or placebo control) o Relative efficacy (i.e., versus an established comparative agent) o Pharmacokinetic behavior o Pharmacoeconomic outcomes o Effects in special populations (e.g., pediatric or renal impaired patients)

3 BioStrategics Consulting Ltd 4. Primary and secondary clinical end points Non-drug-dependent objectives o Natural history of disease o Pilot data to guide the design of subsequent trials Define primary and secondary clinical trial end points. Phase 1: The end points for first in human studies are usually safety and are conducted in human volunteers. The safety outcomes are assessed after single and multiple dose administrations. Phase 2: These studies are usually exploratory studies in each candidate indication to determine which are most suitable for further development. Phase 2 studies often explore the most appropriate dose of drug to use too, looking for the best efficacy without causing undue safety risks. Phase 3: The FDA and the market place requires the demonstration of clinical relevance and benefit. Typically, the end point should reduce mortality, extend longevity or some other clinical benefit and would require the study to follow patients for a longer period of time. Key commercial questions to consider: 1. What is it your drug really does? How do you define it? What is the benefit it offers over existing treatments? For example, do you know or suspect you have an advantage over the standard of care treatment? If so, how do you articulate it? More critically, how to you define it in a way that translates into a clinical trial end point? 2. Exactly what is the market your drug addresses? Which patients does your drug help most? How can this segment be optimally defined for the purposes of clinical trial conduct and, ultimately, the product label? 3. What information would a doctor want to see to convince them to change their prescribing habits? For example, would a comparison study with the standard of care treatment have great influence, even if not necessary for regulatory approval? If so, what is the magnitude of advantage that must be shown to make your drug a relevant competitor? 4. What product profile would convince a patient to demand this drug from their physician? How can the more technical clinical benefit shown in trials be communicated on a lay level? 5. What information would insurance carriers need to convince them to include this drug in their formularies? Could it reduce overall healthcare costs? Can it replace

703 1 of 11 POLICY The Beaumont Research Coordinating Center (BRCC) will provide advice to clinical trial investigators on protocol development, content and format. Upon request, the BRCC will review a

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